Who Should Decide If Gene Therapy Is Too Risky, Patients or the FDA?

BioViva and its controversial CEO Liz Parrish want to bring experimental gene therapies to patients prior to approval by regulators in a quest to cure aging.

Gene therapy is one of those biomedical breakthroughs that has promised to cure Alzheimer’s, cancer, and other disorders for decades. Armed with gene-editing tools like CRISPR, it’s easier than ever to snip out problematic strands of DNA and replace them with new code that deactivates or reverses genetic mutations that cause disease.

Working with mice, researchers have used gene therapy to restore sight to the blind, reprogram the body’s own T cells to attack cancerous tumors, prevent the formation of amyloid plaques in Alzheimer’s brains, and more. But the US Food and Drug Administration and many other international regulatory bodies have been slow to approve human trials due to the high risks associated with tinkering with a patient’s DNA.

Liz Parrish thinks that the FDA and the medical establishment have it backward. Parrish is the founder and CEO of BioViva, a Seattle-based company that is pushing for open access to potentially life-saving gene therapy technology now, not later. The real risk, Parrish argues, is that more than a hundred thousand people die each day, and millions more suffer, while we wait for regulatory agencies to approve drugs that can save lives.

To prove her commitment to the cause, Parrish traveled to an undisclosed clinic in Colombia in 2015 and had herself injected with two gene therapies designed to improve muscle function and lengthen her telomeres, the caps on chromosomes that shorten with age and have been linked with disease-causing genetic malfunctions.

Six months after the procedure, BioViva announced that not only was Parrish alive and well, but her telomeres had grown by nine percent — the equivalent of 20 years — and her muscle mass had increased while intramuscular fat had decreased.

Parrish’s self-experimentation drew criticism from the wider research community for leapfrogging the necessary pre-clinical trials for establishing the safety and efficacy of an experimental therapy.

“Historically, we’ve always moved forward on the backs of pioneers,” Parrish told Seeker. “I didn’t do it to be a hero; I did it to prove that it’s safe. We got more data from that one test than in all the mouse data from the past 20 years and that really can’t be discredited. One human is worth about a billion mice. I just really needed to show that the therapies were safe and we really need to move forward.”

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BioViva and its controversial CEO aren’t interested in playing by the rules of the medical and research establishment. For starters, Parrish believes that the real enemy of human health, the one overriding “disorder” that ultimately drives diseases like Alzheimer’s, heart disease, and cancer is aging itself.

In public presentations and interview, Parrish talks about ten “hallmarks” of biological aging — things like telomere shortening and cellular senescence — that, if treated with gene therapy, could lead to a dramatic increase in human “health span.” The goal isn’t necessarily to live longer, but to live healthier longer, free of age-associated diseases.

BioViva isn’t a research institute and Parrish isn’t a scientist. Instead, the company positions itself as a “translational engine,” a private company bringing together international researchers and physicians to speed the delivery of experimental therapies.

“That means getting drugs to people as soon as possible, with gene therapies at the prominent head of what we do,” said Parrish.

The death of Jesse Gelsinger from an experimental gene therapy in 1999 still casts a shadow over the field.

To make this happen, Parrish said, BioViva is applying to run human clinical trials outside of the United States, including in Africa and “a country in Europe.” It’s also partnering with other companies to open a gene therapy clinic somewhere in Central America where the regulatory regime allows patients to undergo consensual medical treatments that haven’t yet been fully tested.

Jeffrey Kahn, director of the Johns Hopkins Berman Institute of Bioethics, said in an email to Seeker that there are reasons why there are so few FDA-approved gene therapies.

“Historically, they are very risky and have not shown efficacy,” wrote Kahn. “I don’t see how bypassing the oversight and approval process will do anything but expose more people to very significant risks without adequate protections or recourse.”

The death of Jesse Gelsinger from an experimental gene therapy in 1999 still casts a shadow over the field, likely fueling the FDA’s highly cautious position.

Parrish sees things very differently. The only way to truly know if these therapies work in human bodies — as opposed to mice or human cells in the lab — is to give patients the right to decide if the risk is worth it. And the more people who choose to undergo these therapies, the more data BioViva can collect and publish about their true risk and efficacy.

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BioViva’s initial focus will be on treating only the sickest and oldest patients who have exhausted other medical options, but Parrish says that eventually gene therapies will be administered “younger and younger” as a form of preventative medicine.

“We’re looking at a total paradigm shift,” said Parrish. “In 20 years, I believe that we’ll be giving a myriad of these gene therapies to people in their 30s. And that will provide the best defense against the complex diseases of aging. But right now we’ll use them in the sickest patients with the highest need.”

As part of this paradigm shift, Parrish explains, BioViva wants to commercialize its gene therapies more like a technology than a conventional drug. Today, when you get sick, you go to your doctor who gives you a prescription for an FDA-approved medication. Conversely, if you want some new app for your phone or upgraded hardware for your computer, you just go online and buy it. BioViva’s clinics will provide the same kind of on-demand upgrades, but this time to your DNA.

“It will all be done in a safe clinic with medical doctors and emergency staff, of course,” said Parrish, “But it’s literally just going and buying technology as you would buy technology for anything else in your life.”

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